Are We The Source of Knowledge?

We started hearing about it a couple years ago: an ALS member of Patients Like Me had seen (and translated—it was in Italian) a medical conference poster with results showing lithium carbonate could slow the disease’s progression. That study was a single-blind trial of 16 treated patients and 28 controls. The results spread through the ALS community and soon, patients began talking their physicians into prescribing lithium carbonate off-label. PLM soon had 348 members reporting on the effects of their use of the drug.

PLM realized they had an opportunity to study the experience of their members who were—effectively—experimenting with the drug. PLM couldn’t randomize, so they developed an algorithm and matched 149 treated patients to 447 controls based on the progression of their disease course.

On Sunday, the journal Nature Biotechnology published PLM’s findings showing after 12 months of treatment, lithium carbonate had no effect on disease progression. PLM reports that subsequent clinical trials reached similar conclusions.

What’s important here is to recognize the potential to conduct research using patient self-reported data from an online social community. PLM’s sweet spot is social communities for ambiguous diseases (that is, diseases we’re still learning about, diseases that don’t have clear, effective treatment protocols) where the patient does a lot of care at home. To be sure, PLM is a pretty sophisticated community, but it’s intriguing to think about where we might be in 10-15 years.

A couple of us met last week with PLM’s Jamie Heywood and Dave Clifford. We had a ranging discussion—hard to avoid with Heywood—that included linking patient self-reported data with clinicians, conducting research with this data, and business models. A fundamental question Heywood is exploring is “whether it’s faster to get to learning health system through the current confines of the health system or through something like PLM.”

Given the growing ability and inclination of patients to capture and share details on their own experiences, how powerful a role is there for the analysis of this sort of data in our efforts to accelerate the discovery of new treatments for disease?

This commentary originally appeared on the RWJF Pioneering Ideas blog.